BioCentury
ARTICLE | Distillery Therapeutics

Musculoskeletal

March 8, 2017 7:00 PM UTC

Patient sample and mouse studies suggest activating AMPK or inhibiting mTORC1 could help treat DM1. In muscle tissue samples from patients and a genetic mouse model of DM1, levels of phosphorylated RPS6, which is produced by mTORC1 activity, were higher than in samples from healthy volunteers and wild-type mice, respectively. Also in the mouse samples, AMPK pathway activity was lower than in samples from wild-type mice. In the model, an AMPK activator tool compound increased muscle relaxation, and the generic mTOR inhibitor rapamycin decreased levels of phosphorylated RPS6 in muscle tissue, and increased muscle relaxation and muscle force compared with vehicle. Next steps could include testing AMPK activators and mTORC1 inhibitors in other mouse models of DM1.

Betagenon AB has O304, an AMPK activator, in Phase II testing for diabetes and Phase I testing for obesity...